Trial Outcomes & Findings for A Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2 - Protocol B (NCT NCT03319849)

NCT ID: NCT03319849

Last Updated: 2024-09-24

Results Overview

The rate of change in the area of EZ loss (IS/OS; macular photoreceptor loss) from baseline through month 24, as assessed using SD-OCT in the study eye of participants with MacTel.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

119 participants

Primary outcome timeframe

End point timeframe is through Month 24. Baseline, Month 6, 12, 16, 20 and 24. Month 6 was collected but not included in the primary analyses.

Results posted on

2024-09-24

Participant Flow

Screening period for up to 30 days

119 subjects enrolled (6 participants withdrew after randomization) but prior to the implantation/sham surgery procedure; 59 subjects in NT-501 arm and 54 subjects in sham arm.

Unit of analysis: eyes

Participant milestones

Participant milestones
Measure	NT-501 NT-501: Surgery to receive one NT-501 device implant.	Sham Sham: Non-penetrating sham procedure to mimic implant procedure.
Overall Study STARTED	60 60	59 59
Overall Study Treated (Underwent Surgery)	59 59	54 54
Overall Study COMPLETED	57 57	49 49
Overall Study NOT COMPLETED	3 3	10 10

Reasons for withdrawal

Reasons for withdrawal
Measure	NT-501 NT-501: Surgery to receive one NT-501 device implant.	Sham Sham: Non-penetrating sham procedure to mimic implant procedure.
Overall Study Adverse Event	1	0
Overall Study Physician Decision	0	1
Overall Study Withdrawal by Subject	0	1
Overall Study COVID-19	0	1
Overall Study Other	1	2
Overall Study Eligible and did not enroll in amendment	1	3
Overall Study Lost to Follow-up	0	2

Baseline Characteristics

A Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia Type 2 - Protocol B

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	NT-501 n=59 Participants Test product NT-501: Each NT-501 implant consisted of hCNTF-secreting NTC-201-6A.02 cells encapsulated within supportive matrices and surrounded by a semipermeable polymer membrane. The NTC-201-6A cells continuously secrete CNTF from the NT-501 implant into the vitreous cavity. Implanted by a qualified Health Care Professional.	Sham n=54 Participants A sham surgical procedure was performed to mimic the implant procedure; there was no comparator product. Sham Procedure: The sham surgery involved a superficial conjunctival incision performed under local anesthetic and closure with a single suture.	Total n=113 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	42 Participants n=93 Participants	36 Participants n=4 Participants	78 Participants n=27 Participants
Age, Categorical >=65 years	17 Participants n=93 Participants	18 Participants n=4 Participants	35 Participants n=27 Participants
Age, Continuous	58.5 years STANDARD_DEVIATION 7.61 • n=93 Participants	58.7 years STANDARD_DEVIATION 8.87 • n=4 Participants	58.6 years STANDARD_DEVIATION 8.20 • n=27 Participants
Sex: Female, Male Female	46 Participants n=93 Participants	36 Participants n=4 Participants	82 Participants n=27 Participants
Sex: Female, Male Male	13 Participants n=93 Participants	18 Participants n=4 Participants	31 Participants n=27 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	4 Participants n=93 Participants	4 Participants n=4 Participants	8 Participants n=27 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	55 Participants n=93 Participants	49 Participants n=4 Participants	104 Participants n=27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	3 Participants n=93 Participants	1 Participants n=4 Participants	4 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) White	55 Participants n=93 Participants	47 Participants n=4 Participants	102 Participants n=27 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=93 Participants	6 Participants n=4 Participants	7 Participants n=27 Participants
Baseline Ocular Characteristics (mITT) - Ocular Visual Acuity	74.4 Best-corrected visual acuity (letters) STANDARD_DEVIATION 7.76 • n=93 Participants	73.6 Best-corrected visual acuity (letters) STANDARD_DEVIATION 9.23 • n=4 Participants	74.0 Best-corrected visual acuity (letters) STANDARD_DEVIATION 8.47 • n=27 Participants
Baseline Ocular Characteristics (mITT) - Area of EZ loss (mm^2)	0.518 Area of EZ loss (mm^2) STANDARD_DEVIATION 0.3120 • n=93 Participants	0.476 Area of EZ loss (mm^2) STANDARD_DEVIATION 0.2934 • n=4 Participants	0.498 Area of EZ loss (mm^2) STANDARD_DEVIATION 0.3027 • n=27 Participants
Baseline Ocular Characteristics (mITT) - Monocular Reading Speed	96.49 words per minute STANDARD_DEVIATION 47.314 • n=93 Participants	94.09 words per minute STANDARD_DEVIATION 42.806 • n=4 Participants	95.36 words per minute STANDARD_DEVIATION 45.051 • n=27 Participants
Baseline Ocular Characteristics (mITT) - Pupil Diameter	3.53 Pupil diameter (mm) STANDARD_DEVIATION 1.130 • n=93 Participants	3.90 Pupil diameter (mm) STANDARD_DEVIATION 1.092 • n=4 Participants	3.70 Pupil diameter (mm) STANDARD_DEVIATION 1.123 • n=27 Participants
Intraocular Pressure (mmHg)	15.2 mmHg STANDARD_DEVIATION 3.19 • n=93 Participants	14.6 mmHg STANDARD_DEVIATION 2.70 • n=4 Participants	14.9 mmHg STANDARD_DEVIATION 2.97 • n=27 Participants
Natural Lens Present Yes	52 Participants n=93 Participants	46 Participants n=4 Participants	98 Participants n=27 Participants
Natural Lens Present No	7 Participants n=93 Participants	8 Participants n=4 Participants	15 Participants n=27 Participants

PRIMARY outcome

Timeframe: End point timeframe is through Month 24. Baseline, Month 6, 12, 16, 20 and 24. Month 6 was collected but not included in the primary analyses.

Population: The efficacy and safety of the NT-501 implant that delivers a daily dose of CNTF in comparison to sham surgery with no implant, in participants with confirmed MacTel.

The rate of change in the area of EZ loss (IS/OS; macular photoreceptor loss) from baseline through month 24, as assessed using SD-OCT in the study eye of participants with MacTel.

Outcome measures

Outcome measures
Measure	NT-501 n=58 Participants NT-501: Surgery to receive one NT-501 device implant.	Sham n=57 Participants Sham: Non-penetrating sham procedure to mimic implant procedure.	Aggregate Interpolated Retinal Sensitivity Loss (dB) - NT-501 Aggregate Interpolated Retinal Sensitivity Loss (dB) with NT-501 treatment through Month 24	Aggregate Interpolated Retinal Sensitivity Loss (dB) - Sham Aggregate Interpolated Retinal Sensitivity Loss (dB) with sham treatment through Month 24
The Rate of Change in the Area of EZ Area of Loss From Baseline Through Month 24	0.111 mm^2 Standard Error 0.0142	0.160 mm^2 Standard Error 0.0149	—	—

SECONDARY outcome

Timeframe: Baseline through 24 months.

Population: All treated participants (mITT population)

Change from baseline in retinal sensitivity loss as measured by as measured by Macular Integrity Assessment (MAIA)

Outcome measures

Outcome measures
Measure	NT-501 n=59 Participants NT-501: Surgery to receive one NT-501 device implant.	Sham n=54 Participants Sham: Non-penetrating sham procedure to mimic implant procedure.	Aggregate Interpolated Retinal Sensitivity Loss (dB) - NT-501 n=59 Participants Aggregate Interpolated Retinal Sensitivity Loss (dB) with NT-501 treatment through Month 24	Aggregate Interpolated Retinal Sensitivity Loss (dB) - Sham n=54 Participants Aggregate Interpolated Retinal Sensitivity Loss (dB) with sham treatment through Month 24
Mean Change in Aggregate Retinal Sensitivity Loss and Aggregate Interpolated Retinal Sensitivity Loss by Microperimetry (mITT Population) Baseline Actual	55.54 dB Standard Deviation 56.050	51.84 dB Standard Deviation 57.380	34864.17 dB Standard Deviation 35305.260	32445.03 dB Standard Deviation 38667.790
Mean Change in Aggregate Retinal Sensitivity Loss and Aggregate Interpolated Retinal Sensitivity Loss by Microperimetry (mITT Population) Month 24 Change from Baseline	40.02 dB Standard Deviation 51.278	41.97 dB Standard Deviation 41.111	24859.88 dB Standard Deviation 31165.862	26309.32 dB Standard Deviation 25374.040
Mean Change in Aggregate Retinal Sensitivity Loss and Aggregate Interpolated Retinal Sensitivity Loss by Microperimetry (mITT Population) Month 24 Actual	97.77 dB Standard Deviation 78.620	93.02 dB Standard Deviation 70.414	61076.49 dB Standard Deviation 52619.379	58360.26 dB Standard Deviation 50535.070

SECONDARY outcome

Timeframe: Baseline through 24 months.

Population: All treated participants (mITT population) without missing data

Change from baseline through Month 24 for Monocular reading speed assessed using International Reading Speed Texts (IReST) cards developed by the IReST Study Group 21

Outcome measures

Outcome measures
Measure	NT-501 n=59 Participants NT-501: Surgery to receive one NT-501 device implant.	Sham n=54 Participants Sham: Non-penetrating sham procedure to mimic implant procedure.	Aggregate Interpolated Retinal Sensitivity Loss (dB) - NT-501 Aggregate Interpolated Retinal Sensitivity Loss (dB) with NT-501 treatment through Month 24	Aggregate Interpolated Retinal Sensitivity Loss (dB) - Sham Aggregate Interpolated Retinal Sensitivity Loss (dB) with sham treatment through Month 24
Monocular Reading Speed (mITT Population) Baseline Actual	96.49 Reading Speed (words per minute) Standard Deviation 47.314	94.09 Reading Speed (words per minute) Standard Deviation 42.806	—	—
Monocular Reading Speed (mITT Population) Month 24 Actual	92.18 Reading Speed (words per minute) Standard Deviation 45.090	77.33 Reading Speed (words per minute) Standard Deviation 43.899	—	—
Monocular Reading Speed (mITT Population) Month 24 Change from Baseline	-5.46 Reading Speed (words per minute) Standard Deviation 29.648	-18.88 Reading Speed (words per minute) Standard Deviation 33.705	—	—

Adverse Events

NT-501

Serious events: 11 serious events

Other events: 56 other events

Deaths: 1 deaths

Sham

Serious events: 10 serious events

Other events: 48 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

CMO

Neurotech Pharmaceuticals, LLC

Phone: 401-333-3880

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Site agrees not to make any publication prior to Sponsor's initial publication. Site agrees that proposed manuscripts, abstracts, or oral presentations relating to the Study Drug will be submitted directly to Sponsor at least sixty (60) days prior to submission for publication or presentation to ensure that Confidential Information (including Clinical Data or Study results not disclosed in Sponsor's prior publication), Sponsor Technology or Sponsor-Owned Inventions are protected.
Publication restrictions are in place

Restriction type: OTHER

Measure	NT-501 n=59 participants at risk NT-501: Surgery to receive one NT-501 device implant.	Sham n=54 participants at risk Sham: Non-penetrating sham procedure to mimic implant procedure.
Injury, poisoning and procedural complications Suture related complication	5.1% 3/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	0.00% 0/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Cardiac disorders Angina pectoris	1.7% 1/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	0.00% 0/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Nervous system disorders Transient ischaemic attack	3.4% 2/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	0.00% 0/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Nervous system disorders Cerebrovascular accident	1.7% 1/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	0.00% 0/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Reproductive system and breast disorders Endometriosis	1.7% 1/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	0.00% 0/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Respiratory, thoracic and mediastinal disorders Chronic obstructive pulmonary disease	1.7% 1/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Respiratory, thoracic and mediastinal disorders Respiratory failure	1.7% 1/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	0.00% 0/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Endocrine disorders Goitre	1.7% 1/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	0.00% 0/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Product Issues Device extrusion	1.7% 1/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	0.00% 0/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Infections and infestations Cellulitis	1.7% 1/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Infections and infestations Osteomyelitis	1.7% 1/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	0.00% 0/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Breast cancer	0.00% 0/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Colon cancer	0.00% 0/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Invasive ductal breast carcinoma	0.00% 0/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Injury, poisoning and procedural complications Tendon rupture	0.00% 0/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Cardiac disorders Atrial fibrillation	0.00% 0/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Cardiac disorders Palpitations	0.00% 0/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Gastrointestinal disorders Small intestinal obstruction	0.00% 0/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Psychiatric disorders Depression	0.00% 0/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Musculoskeletal and connective tissue disorders Intervertebral disc protrusion	0.00% 0/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Musculoskeletal and connective tissue disorders Osteoarthritis	0.00% 0/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.
Infections and infestations COVID-19	0.00% 0/59 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.	1.9% 1/54 • During the Clinical Trial Any non-ocular events and/or ocular events in the study eye.